Vasa Therapeutics Announces Positive Topline Results from Randomized, Placebo-Controlled Phase 1c Trial of VS-041 for Treatment of Heart Failure with Preserved Ejection Fraction (HFpEF)

• Significant reduction of NT-proBNP after a 28-day treatment with VS-041, a novel therapeutic that targets fibroinflammation and abnormal turnover of extracellular matrix in the myocardium.
• VS-041, an oral inhibitor of narrow spectrum of matrix metalloproteinases, was safe and well-tolerated in the affected population
• Vasa's trial pioneered use of an elevated serum endotrophin level to identify an endotype of HFpEF patients to improve efficiency of future clinical trials

ENCINITAS, Calif. and WROCLAW, Poland, Nov. 12, 2026 /PRNewswire/ -- Vasa Therapeutics ("Vasa"), a private, clinical stage biotechnology company developing novel therapies for cardiovascular and metabolic aging, today announced positive results from a randomized, placebo-controlled Phase 1c trial of its lead investigational therapy, VS-041, a narrow spectrum small molecule inhibitor of matrix metalloproteinases (MMPs) for the treatment of heart failure with preserved ejection fraction (HFpEF) demonstrating a clinical Proof-of-Concept in the affected population.

VS-041 achieved a reduction in NT-proBNP, an important biomarker of cardiac wall stress and heart failure in patients receiving background guideline-directed medical therapy for HFpEF. At two doses of treatment, VS-041 was safe and well-tolerated. The study also supported the utility of using serum endotrophin, as measured by the NordicProC6 assay, to identify a more homogenous subpopulation of patients with fibroinflammation as the likely cause of diastolic dysfunction.

Ongoing analyses of exploratory clinical measures and markers of fibroinflammation and collagen turnover in the extracellular matrix (ECM) will be presented in early 2027. 

VS-041 was demonstrated to ameliorate cardiac fibrosis and improve diastolic function in preclinical models of HFpEF.  In healthy participant Phase 1 clinical trial, VS-041 was safe and well-tolerated at doses commensurate with preclinical efficacy. VS-041 was awarded Fast Track Designation by the Food and Drug Administration in November 2025. 

"Vasa had three important goals for the recently completed Phase 1c trial in patients with HFpEF.  First, we showed that narrow-spectrum MMP inhibition is safe and well tolerated in a population of HFpEF patients with increased risk of unfavorable outcomes, and the reduction in NT-proBNP, achieved within 28 days suggests a compelling path forward in the clinic.  Secondly, we demonstrated that serum endotrophin can be used prospectively to identify an endotype of HFpEF patients that cannot be characterized by clinical data alone. This novel enrichment strategy should translate to smaller and potentially faster development.  And lastly, we are learning about the impact of VS-041 on fibroinflammation in the ECM, an important pathophysiology underlying HFpEF, chronic kidney disease, and cardiorenal metabolic syndrome," said Artur Plonowski, MD, PhD, Chief Executive Officer and co-founder of Vasa Therapeutics.  "I want to thank the patients and investigators who made this novel study possible and helped Vasa achieve this critical milestone on our mission of advancing innovative therapies for cardiovascular diseases."

About VS-041

VS-041 is an oral compound that was discovered and developed by Vasa for the potential treatment of HFpEF and other chronic diseases characterized by fibroinflammation, such as hypertrophic cardiomyopathy and chronic kidney disease.  Development of VS- 041 was co-funded by the European Regional Development Fund and the Polish National Centre for Research and Development (POIR.01.01.01-00-1210/10-01).

About Heart Failure with Preserved Ejection Fraction (HFpEF)

HFpEF is a complex and progressive form of heart failure characterized by stiffening and fibroinflammation of the heart muscle and impaired relaxation despite normal contractile function. HFpEF affects ~3 million individuals in the US and ~10 million globally. Currently approved therapies for HFpEF provide clinical improvement but do not result in disease modification and durable improvement upon discontinuation of the treatment.

About Vasa Therapeutics

Vasa Therapeutics is a clinical-stage biopharmaceutical company developing novel therapeutics that target the pathophysiology of cardiovascular and muscle aging.  The company has developed a pipeline of four unique assets targeting unmet medical needs in heart failure, sarcopenia, rare neuromuscular diseases, peripheral arterial disease and life-threatening arrhythmias. For more information, please visit vasatherapeutics.com

SOURCE Vasa Therapeutics